米国の希少疾病用医薬品（オーファンドラッグ）市場：治療分野別、薬剤タイプ別、流通チャネル別、地域別、機会、予測、2018年～2032年

米国の希少疾病用医薬品（オーファンドラッグ）の市場規模は、2025年～2032年の予測期間中に10.25%のCAGRで拡大し、2024年の903億9,000万米ドルから2032年には1,973億1,000万米ドルに成長すると予測されています。米国の希少疾病用医薬品市場は、希少疾病の蔓延率の上昇と診断の強化により力強い成長を遂げています。市場独占権や税額控除といった政府の優遇措置が医薬品の技術革新を後押ししています。さらに、がん領域が依然として最大セグメントであり、希少がん治療への注目の高まりが市場拡大を加速させています。

例えば、2024年9月11日、希少疾患の治療開発に専念する細胞代謝とPK活性化のフロントランナーであるAgios Pharmaceuticals, Inc.は、骨髄異形成症候群（MDS）の治療を目的とした革新的なピルビン酸キナーゼ（PK）活性化薬であるテバピバット（AG-946）が米国食品医薬品局（FDA）からオーファンドラッグに指定されたと発表しました。

当レポートでは、米国の希少疾病用医薬品（オーファンドラッグ）市場について調査し、市場の概要とともに、治療分野別、薬剤タイプ別、流通チャネル別、地域別動向、および市場に参入する企業のプロファイルなどを提供しています。

目次

第1章 プロジェクトの範囲と定義

第2章 調査手法

第3章 米国の関税の影響

第4章 エグゼクティブサマリー

第5章 米国のオーファンドラッグ市場の見通し、2018年～2032年

• 市場規模分析と予測
• 市場シェア分析と予測
  • 治療分野別
    • 腫瘍学
    • 血液学
    • 神経学
    • 内分泌学
    • 心血管系
    • 呼吸器
    • 免疫療法
    • 感染症
    • その他
  • 薬剤タイプ別
    • 生物学的製剤
    • 非生物学的製剤
  • 流通チャネル別
    • 病院薬局
    • 小売薬局とドラッグストア
    • オンライン薬局
  • 地域別
    • 北西部
    • 南西部
    • 西部
    • 南東部
    • 中西部
  • 企業別市場シェア分析（上位5社およびその他- 金額別、2024年）
• 2024年の市場マップ分析

第6章 需要供給分析

第7章 バリューチェーン分析

第8章 ポーターのファイブフォース分析

第9章 PESTLE分析

第10章 価格分析

第11章 市場力学

• 市場の促進要因
• 市場の課題

第12章 市場動向と発展

第13章 規制枠組みとイノベーション

• 規制当局の承認
• 臨床試験

第14章 特許の情勢

第15章 ケーススタディ

第16章 競合情勢

• 市場リーダートップ5の競合マトリックス
• 参入企業トップ5のSWOT分析
• 市場の主要企業トップ10の情勢
  • Novartis AG
  • Amgen, Inc.
  • Bristol-Myers Squibb Company
  • AstraZeneca plc
  • Pfizer Inc.
  • Novo Nordisk A/S
  • Sanofi S.A.
  • Johnson & Johnson Services, Inc.
  • F. Hoffmann-La Roche Ltd
  • Vertex Pharmaceuticals Incorporated

第17章 戦略的提言

第18章 調査会社について・免責事項

List of Tables

• Table 1. Competition Matrix of Top 5 Market Leaders
• Table 2. Mergers & Acquisitions/ Joint Ventures (If Applicable)
• Table 3. About Us - Regions and Countries Where We Have Executed Client Projects

List of Figures

• Figure 1. United States Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 2. United States Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 3. United States Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 4. United States Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 5. United States Orphan Drugs Market Share (%), By Region, 2018-2032F
• Figure 6. By Therapy Area Map-Market Size (USD Billion) & Growth Rate (%), 2024
• Figure 7. By Drug Type Map-Market Size (USD Billion) & Growth Rate (%), 2024
• Figure 8. By Distribution Channel Map-Market Size (USD Billion) & Growth Rate (%), 2024
• Figure 9. By Region Map-Market Size (USD Billion) & Growth Rate (%), 2024

United States orphan drugs market is projected to witness a CAGR of 10.25% during the forecast period 2025-2032, growing from USD 90.39 billion in 2024 to USD 197.31 billion in 2032. The U.S. orphan drugs market is witnessing strong growth due to rising rare disease prevalence and enhanced diagnostics. Government incentives like market exclusivity and tax credits are encouraging pharmaceutical innovation. Additionally, oncology remains the largest segment, with increasing focus on rare cancer therapies accelerating market expansion.

For instance, on September 11, 2024, Agios Pharmaceuticals, Inc., a frontrunner in cellular metabolism and PK activation dedicated to developing therapies for rare diseases, announced that the U.S. Food and Drug Administration (FDA) has awarded orphan drug designation to its innovative pyruvate kinase (PK) activator, tebapivat (AG-946), intended for the treatment of myelodysplastic syndromes (MDS).

Rising Prevalence of Rare Diseases Fueling Demand for Orphan Drugs

The rising incidence of rare diseases in the United States has markedly increased the demand for orphan drugs. The National Institutes of Health (NIH) reports that around 7,000 rare diseases impact more than 30 million Americans. Many of these conditions lack adequate treatment options, leading to a significant unmet need for specialized therapeutics. Orphan drugs are designed specifically for these diseases, and their market has expanded due to heightened awareness, earlier diagnoses, and advancements in genetic testing. Additionally, the U.S. Food and Drug Administration (FDA) has introduced various incentives, including tax credits, market exclusivity, and accelerated approval processes, to promote the development of orphan drugs. These measures have led to a rise in research and development investments from pharmaceutical companies. For instance, in February 2024, Amgen received FDA approval for BLINCYTO (blinatumomab), an orphan drug for the treatment of minimal residual disease (MRD) in acute lymphoblastic leukemia (ALL) patients. This approval not only adds to Amgen's oncology pipeline but also highlights the industry's growing commitment to addressing rare disease burdens.

Government Incentives and Regulatory Support Accelerate Orphan Drug Approvals

The proactive regulatory framework established by the U.S. government significantly drives the orphan drug market. The Orphan Drug Act of 1983 provided essential regulatory and financial incentives, including seven years of market exclusivity, research grants, and tax credits for clinical trials. These advantages motivate biotech and pharmaceutical companies to pursue the development of treatments for rare diseases, even with limited patient populations. The FDA's Office of Orphan Products Development (OOPD) is instrumental in accelerating these therapies' review and approval processes through initiatives such as Fast Track, Breakthrough Therapy, and Priority Review designations. In recent years, this supportive environment has resulted in a notable increase in orphan drug designations and approvals. A notable example is when Acadia Pharmaceuticals Inc. had declared in the U.S. in March 2023, that the U.S. Food and Drug Administration (FDA) had approved DAYBU (trofinetide) to treat Rett syndrome in both adult and pediatric patients aged two years and older. DAYBUE is the sole medication authorized to treat Rett syndrome. The approval underlines how streamlined pathways and strong regulatory backing are helping more orphan drugs reach patients faster, boosting innovation and accessibility in the U.S. market.

Oncology Segment Dominates with Increasing Development of Rare Cancer Therapies

Oncology represents the largest U.S. orphan drugs market segment among various therapeutic areas. The increasing prevalence of rare cancers, including soft tissue sarcomas, gliomas, and specific blood malignancies, has notably heightened the demand for specialized treatment options. Pharmaceutical firms are sharpening their focus on developing targeted therapies and immuno-oncology medications that provide enhanced effectiveness and reduced side effects compared to traditional treatments. The robust presence of biotechnology companies, investments in precision medicine, and a growing pipeline of clinical trials in the U.S. further bolstered this sector's growth. Additionally, the orphan drug designation for oncology products frequently results in expedited regulatory approvals, lower development expenses, and prolonged market exclusivity, rendering it a highly profitable domain. For instance, in August 2023, Bristol Myers Squibb received FDA approval for REBLOZYL (luspatercept-aamt) as a first-line treatment for anemia in adults with lower-risk myelodysplastic syndromes, which includes certain rare cancers. This approval under the Orphan Drug Act highlights the market's strong momentum in rare oncology therapeutics.

Future Market Scenario (2025-2032F)

The orphan drugs market in the United States is set for significant expansion, driven by a growing awareness of rare diseases, favorable regulatory frameworks, and vigorous research and development efforts by pharmaceutical firms. Innovations in genomics and personalized medicine facilitate the discovery of new therapeutic targets, while expedited FDA approval processes are shortening the time required for orphan-designated drugs to reach the market. Additionally, the increasing presence of patient advocacy organizations and advancements in diagnostics are enhancing access to therapies for rare diseases. The prevalence of biologics and heightened investments in gene and cell therapies will likely transform the treatment landscape. With major industry players actively working on innovative pipeline projects, the market is expected to maintain its momentum, providing renewed hope for underrepresented patient populations across diverse therapeutic fields.

Key Players Landscape and Outlook

The key players in the market are significantly investing in the development of orphan drugs and are utilizing strategies such as mergers, acquisitions, partnerships, and new product launches to improve their services and competitiveness. Such efforts will propel significant growth in the market, allowing large-cap industry players to increase their presence and, therefore, find new opportunities in this market.

For instance, Rapid Commercialization Partners (RCP) has joined forces with Orphan Now to assist companies in accelerating the development of orphan drugs. This collaboration seeks to facilitate market entry, thereby providing quicker access to essential treatments for patients globally. The initiative improves the efficiency of commercialization, offering advantages to developers of drugs for rare diseases.

Table of Contents

1. Project Scope and Definitions

2. Research Methodology

3. Impact of U.S. Tariffs

4. Executive Summary

5. United States Orphan Drugs Market Outlook, 2018-2032F

• 5.1. Market Size Analysis & Forecast
  • 5.1.1. By Value
• 5.2. Market Share Analysis & Forecast
  • 5.2.1. By Therapy Area
    • 5.2.1.1. Oncology
    • 5.2.1.2. Hematology
    • 5.2.1.3. Neurology
    • 5.2.1.4. Endocrinology
    • 5.2.1.5. Cardiovascular
    • 5.2.1.6. Respiratory
    • 5.2.1.7. Immunotherapy
    • 5.2.1.8. Infectious Diseases
    • 5.2.1.9. Others
  • 5.2.2. By Drug Type
    • 5.2.2.1. Biologics
    • 5.2.2.2. Non-Biologics
  • 5.2.3. By Distribution Channel
    • 5.2.3.1. Hospital Pharmacies
    • 5.2.3.2. Retail Pharmacies and Drug Stores
    • 5.2.3.3. Online Pharmacies
  • 5.2.4. By Region
    • 5.2.4.1. Northwest
    • 5.2.4.2. Southwest
    • 5.2.4.3. West
    • 5.2.4.4. Southeast
    • 5.2.4.5. Midwest
  • 5.2.5. By Company Market Share Analysis (Top 5 Companies and Others - By Value, 2024)
• 5.3. Market Map Analysis, 2024
  • 5.3.1. By Therapy Area
  • 5.3.2. By Drug Type
  • 5.3.3. By Distribution Channel
  • 5.3.4. By Region

6. Demand Supply Analysis

7. Value Chain Analysis

8. Porter's Five Forces Analysis

9. PESTLE Analysis

10. Pricing Analysis

11. Market Dynamics

• 11.1. Market Drivers
• 11.2. Market Challenges

12. Market Trends and Developments

13. Regulatory Framework and Innovation

• 13.1. Regulatory Approvals
• 13.2. Clinical Trials

14. Patent Landscape

15. Case Studies

16. Competitive Landscape

• 16.1. Competition Matrix of Top 5 Market Leaders
• 16.2. SWOT Analysis for Top 5 Players
• 16.3. Key Players Landscape for Top 10 Market Players
  • 16.3.1. Novartis AG
    • 16.3.1.1. Company Details
    • 16.3.1.2. Key Management Personnel
    • 16.3.1.3. Products and Services
    • 16.3.1.4. Financials (As Reported)
    • 16.3.1.5. Key Market Focus and Geographical Presence
    • 16.3.1.6. Recent Developments/Collaborations/Partnerships/Mergers and Acquisitions
  • 16.3.2. Amgen, Inc.
  • 16.3.3. Bristol-Myers Squibb Company
  • 16.3.4. AstraZeneca plc
  • 16.3.5. Pfizer Inc.
  • 16.3.6. Novo Nordisk A/S
  • 16.3.7. Sanofi S.A.
  • 16.3.8. Johnson & Johnson Services, Inc.
  • 16.3.9. F. Hoffmann-La Roche Ltd
  • 16.3.10. Vertex Pharmaceuticals Incorporated

Companies mentioned above DO NOT hold any order as per market share and can be changed as per information available during research work.

17. Strategic Recommendations

18. About Us and Disclaimer